Gene Therapy Developed To Boost Myelin Repair

Demyelinating diseases like multiple sclerosis are known to be a result of damage to the insulating material around nerve cells called myelin. Such diseases may cause not only the myelin to become damaged in the process but also damage the specialized cells whose function is to repair this important insulation material for nerve cells. This causes the myelin damage to further worsen, causing the nerve cells to send and receive signals inefficiently that leads to a number of physical symptoms such as deteriorating muscle control and coordination, numbness, blurred vision and others. But now, researchers may have found a potential means to help repair the damaged myelin in the brain.

Researchers from the California Institute of Technology or Caltech have developed a form of gene therapy that has proven to be successful in promoting remyelination in a mouse model of MS. The findings are published in The Journal of Neuroscience. The said therapy aims to help the brain to replace damaged myelin and oligodendrocytes, the specialized cells that help repair myelin.

The said therapy makes use of the leukemia inhibitory factor or LIF in promoting the process of remyelination. LIF is a naturally occurring protein that is known to promote the self-renewal of neural stem cells that helps in myelin repair as well as help reduce the attacks caused by the body’s immune cells to myelin as seen from mouse models of MS.

According to Benjamin Deverman, a post-doctoral fellow in biology at Caltech and lead author of the study, “We’ve developed a gene therapy to stimulate production of new oligodendrocytes from stem and progenitor cells – both of which can become more specialized cell types – that are resident in the adult central nervous system. In other words, we’re using the brain’s own progenitor cells as a way to boost repair.”

Although gene therapy has already been a focus for treatment of MS, what has not yet been done before this said study was to use it in order to stimulate cells in the brain to remyelinate. According to what the researchers have found so far, LIF is vital to stimulating oligodendrocyte progenitor cells to flourish and create new oligodendrocytes. The brain is known to have the ability to produce oligodendrocytes. But it hasn’t been always able to produce enough amounts of such cells in order to mount an effective repair response after demyelination.

“Researchers had been skeptical that a single factor could lead to remyelination of damaged cells,” Deverman stated. “It was thought that you could use factors to stimulate the division and expansion of the progenitor population, and then add additional factors to direct those progenitors to turn into the mature myelin-forming cells. But in our mouse model, when we give our LIF therapy, it both stimulates the proliferation of the progenitor cells and allows them to differentiate into mature oligodendrocytes,” he further added.

The researchers found that LIF caused a very strong response that the treated brain’s levels of myelin-producing oligodendrocytes were restored to that known for healthy populations. The researchers also noted that delivering LIF directly into the brain prevents the potential side effects of the said treatment when it is otherwise infused via the bloodstream.

The next step for the researchers is to conduct human clinical trials by building better vectors for the delivery of LIF into the brain. The researchers are also planning to test the newly developed therapy into additional MS mouse models.

Source: Medical News Today

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